Arimoclomol: a potential therapy under development for ALS

Bibliography

• Rowland LP, Shneider NA Amyotrophic lateral sclerosis. N Engl J Med 2001;344:1688-700
   PubMed Web of Science ®Google Scholar
• Shaw PJ. Science, medicine, and the future: Motor neurone disease. BMJ 1999;318(7191):1118-21
   PubMed Web of Science ®Google Scholar
• Hadano S, Hand CK, Osuga H, A gene encoding a putative GTPase regulator is mutated in familial amyotrophic lateral sclerosis 2. Nat Genet 2001;29:166-73
   PubMed Web of Science ®Google Scholar
• Kanekura K, Hashimoto Y, Niikura T, Alsin, the Product of ALS2 Gene, Suppresses SOD1 Mutant Neurotoxicity through RhoGEF Domain by Interacting with SOD1 Mutants. J Biol Chem 2004;279(18):19247-56
   PubMed Web of Science ®Google Scholar
• Chen YZ, Bennett CL, Huynh HM, DNA/RNA Helicase Gene Mutations in a Form of Juvenile Amyotrophic Lateral Sclerosis (ALS4). Am J Hum Genet 2004;74(6):1128-35
   PubMed Web of Science ®Google Scholar
• Puls I, Jonnakuty C, LaMonte BH, Mutant dynactin in motor neuron disease. Nat Genet 2003;33(4):455-6
   PubMed Web of Science ®Google Scholar
• Rainier S, Bui M, Mark E, Neuropathy Target Esterase Gene Mutations Cause Motor Neuron Disease. Am J Hum Gene 2008;82(3):780-5
   PubMed Web of Science ®Google Scholar
• Valdmanis PN, Daoud H, Dion PA, Rouleau GA. Recent advances in the genetics of amyotrophic lateral sclerosis. Curr Neurol Neurosci Rep 2009;9(3):198-205
   Google Scholar
• Nishimura AL, Mitne-Neto M, Silva HC, A Mutation in the Vesicle-Trafficking Protein VAPB Causes Late-Onset Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis. Am J Hum Genet 2004;75(5):822-31
   PubMed Web of Science ®Google Scholar
• Cid C, Alvarez-Cermeño JC, Regidor I, Low concentrations of glutamate induce apoptosis in cultured neurons: Implications for amyotrophic lateral sclerosis. J Neurol Sci 2003;206(1):91-5
   PubMed Web of Science ®Google Scholar
• Rakhit R, Crow JP, Lepock JR, Monomeric Cu,Zn-superoxide Dismutase Is a Common Misfolding Intermediate in the Oxidation Models of Sporadic and Familial Amyotrophic Lateral Sclerosis. J Biol Chem 2004;279(15):15499-504
   PubMed Web of Science ®Google Scholar
• Ferrante RJ, Browne SE, Shinobu LA, Evidence of Increased Oxidative Damage in Both Sporadic and Familial Amyotrophic Lateral Sclerosis. J Neurochemi 1997;69(5):2064-74
   PubMed Web of Science ®Google Scholar
• Wood JD. Protein aggregation in motor neurone disorders. Neuropathol Appl Neurobiol 2003;29(6):529-45
   PubMed Web of Science ®Google Scholar
• Shibata N, Hirano A, Kobayashi A, Immunohistochemical demonstration of Cu/Zn superoxide dismutase in the spinal cord of patients with familial amyotrophic lateral sclerosis. Acta Histochem Cytochem 1993;26:619-22
   Web of Science ®Google Scholar
• Shibata N, Hirano A, Kobayashi M, Presence of Cu/Zn superoxide dismutase (SOD) immunoreactivity in neuronal hyaline inclusions in spinal cords from mice carrying a transgene for Gly93Ala mutant human Cu/Zn SOD. Acta Neuropathologica 1998;95(2):136-42
   PubMed Web of Science ®Google Scholar
• Shinder GA, Lacourse, MC, Minotti, S, Durham, H.D Mutant Cu/Zn- superoxide dismutase proteins have altered solubility and interact with heat shock/stress proteins in models of amyotrophic lateral sclerosis. J Biol Chem 2001;276:12791–6
   PubMed Web of Science ®Google Scholar
• Bence NF, Sampat RM, Kopito RR. Impairment of the Ubiquitin-Proteasome System by Protein Aggregation. Science 2001;292(5521):1552-5
   PubMed Web of Science ®Google Scholar
• Bruening W, Roy J, Giasson B, Up-Regulation of Protein Chaperones Preserves Viability of Cells Expressing Toxic Cu/Zn-Superoxide Dismutase Mutants Associated with Amyotrophic Lateral Sclerosis. J Neurochem 1999;72(2):693-9
   PubMed Web of Science ®Google Scholar
• Maatkamp A, Vlug A, Haasdijk E, Decrease of Hsp25 protein expression precedes degeneration of motoneurons in ALS-SOD1 mice. Eur J Neurosci 2004;20:14-28
   PubMed Web of Science ®Google Scholar
• Kieran D, Kalmar B, Dick JR, Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice. Nat Med 2004; 10(4):402-5
   Google Scholar
• Cudkowicz ME, Shefner JM, Simpson E, Arimoclomol at dosages up to 300 mg/day is well tolerated and safe in amyotrophic lateral sclerosis. Muscle Nerve 2008;38(1):837-44
   PubMed Web of Science ®Google Scholar
• Amyotrophic Lateral Sclerosis/Riluzole Study Group II: Lacomblez L, Bensimon G, Leigh PN, . Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Lancet 1996;347(9013):1425-31
   PubMed Web of Science ®Google Scholar
• Lechtzin N, Scott Y, Busse AM, Early use of non-invasive ventilation prolongs survival in subjects with ALS. Amyotroph Lateral Scler 2007;8(3):185-8
   PubMed Web of Science ®Google Scholar
• Andersen PM, Borasio GD, Dengler R, Good practice in the management of amyotrophic lateral sclerosis: Clinical guidelines. An evidence-based review with good practice points. EALSC Working Group. Amyotroph Lateral Scler 2007;8(4):195-213
   PubMed Web of Science ®Google Scholar
• Hargitai J, Lewis H, Boros I, Bimoclomol, a heat shock protein co-inducer, acts by the prolonged activation of heat shock factor-1. Biochem Biophys Res Commun 2003;307(3):689-95
   PubMed Web of Science ®Google Scholar
• Lindquist S. The heat-shock response. Ann Rev Biochem 1986;55(1):1151-91
   PubMedGoogle Scholar
• Nanasi PP, Jednakovits A. Multilateral in vivo and in vitro protective effects of the novel heat shock protein coinducer, bimoclomol: Results of preclinical studies. Cardiovasc Drug Rev 2001;19:133-51
   PubMed Web of Science ®Google Scholar
• Kürthy M, Mogyorósi T, Nagy K, Effect of BRX-220 against peripheral neuropathy and insulin resistance in diabetic rat models. Ann Acad Sci 2002;967:482-9
   PubMed Web of Science ®Google Scholar
• Visy J, Fitos I, Mady G, Enantioselective plasma protein binding of bimoclomol. Chirality. 2002;14(8):638-42
   PubMed Web of Science ®Google Scholar
• Brar BK, Stephanou A, Wagstaff MJ, Heat shock proteins delivered with a virus vector can protect cardiac cells against apoptosis as well as against thermal or hypoxic stress. J Mol Cell Cardiol 1999;31(1):135-46
   PubMed Web of Science ®Google Scholar
• Batulan Z, Shinder GA, Minotti S, High threshold for induction of the stress response in motor neurons is associated with failure to activate HSF1. J Neurosci 2003:23(13):5789-98
   PubMed Web of Science ®Google Scholar
• Kalmar B, Novoselov S, Gray A, Late stage treatment with arimoclomol delays disease progression and prevents protein aggregation in the SOD1G93A mouse model of ALS. J Neurochem 2008;107(2):339-50
   PubMed Web of Science ®Google Scholar
• Estévez AG, Crow JP, Sampson JB, Induction of nitric oxide-dependent apoptosis in motor neurons by zinc-deficient superoxide dismutase. Science 1999;286(5449):2498-500
   PubMed Web of Science ®Google Scholar
• Watanabe M, Dykes-Hoberg M, Culotta VC, Histological evidence of protein aggregation in mutant SOD1 transgenic mice and in amyotrophic lateral sclerosis neural tissues. Neurobiol Dis 2001;8(6):933-41
   PubMed Web of Science ®Google Scholar
• Ferrante RJ, Browne SE, Shinobu LA, Evidence of increased oxidative damage in both sporadic and familial ALS. J Neurochem 1997;69(5):2064-74
   PubMed Web of Science ®Google Scholar
• Piao YS, Wakabayashi K, Kakita A, Neuropathology with Clinical Correlations of Sporadic Amyotrophic Lateral Sclerosis: 102 Autopsy Cases Examined Between 1962 and 2000. Brain Pathol 2003;13(1):10-22
   PubMed Web of Science ®Google Scholar
• Johnson BS, Snead D, Lee JJ, TDP-43 is intrinsically aggregation-prone and ALS-linked mutations accelerate aggregation and increase toxicity. J Biol Chem 2009;284(30):20329-39
   PubMed Web of Science ®Google Scholar
• Neumann M, Kwong LK, Lee EB, Phosphorylation of S409/410 of TDP-43 is a consistent feature in all sporadic and familial forms of TDP-43 proteinopathies. Acta Neuropathol 2009;117(2):137-49
   PubMed Web of Science ®Google Scholar
• Kalmar B, Burnstock G, Vrbová G, Upregulation of Heat Shock Proteins Rescues Motoneurones from Axotomy-Induced Cell Death in Neonatal Rats. Exp Neurol 2002;176(1):87-97
   PubMed Web of Science ®Google Scholar
• CytRx Corporation: Unpublished Data
   Google Scholar
• Elman LB, Siderowf A, Houseman G, Venous thrombosis in an ALS population over four years. Amyotroph Lateral Scle 2005;6(4):246-9
   Web of Science ®Google Scholar
• Qureshi MM, Cudkowicz ME, Zhang H, Raynor E. Increased incidence of deep venous thrombosis in ALS. Neurology 2007;68(1):76-7
   PubMed Web of Science ®Google Scholar
• Lacomblez L, Bensimon G, Leigh PN, Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Lancet 1996;347(May 25):1425-31
   PubMedGoogle Scholar
• Miller RG, Moore DH, Gelinas DF, Phase III randomized trial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology 2001;56:843-8
   PubMed Web of Science ®Google Scholar
• Cudkowicz ME, Shefner JM, Schoenfeld DA, A randomized, placebo-controlled trial of topiramate in amyotrophic lateral sclerosis. Neurology 2003;61(4):456-64
   PubMed Web of Science ®Google Scholar
• Gredal O, Werdelin L, Bak S, A clinical trial of dextromethorphan in amyotrophic lateral sclerosis. Acta Neurol Scand 1997;96(1):8-13
   PubMed Web of Science ®Google Scholar
• Norris FH Jr, Calanchini PR, Fallat RJ, The administration of guanidine in amyotrophic lateral sclerosis. Neurology 1974;24(8):721-8
   PubMed Web of Science ®Google Scholar
• Testa D, Caraceni T, Fetoni V. Branched-chain amino acids in the treatment of amyotrophic lateral sclerosis. Neurol 1989;236(8):445-7
   Web of Science ®Google Scholar
• Blin O, Pouget J, Aubrespy G, A double-blind placebo-controlled trial of L-threonine in amyotrophic lateral sclerosis. J Neurol 1992;239(2):79-81
   PubMed Web of Science ®Google Scholar
• Miller RG, Shepherd R, Dao H, Controlled trial of nimodipine in amyotrophic lateral sclerosis. Neuromuscul Disord 1996;6(2):101-4
   PubMed Web of Science ®Google Scholar
• Szczudlik A, Tomik B, Słowik A, Kasprzyk K. Assessment of the efficacy of treatment with pimozide in patients with amyotrophic lateral sclerosis. Introductory notes. Neurol Neurochir Pol 1998;32(4):821-9 [Article in Publish]
   PubMedGoogle Scholar
• Aisen ML, Sevilla D, Edelstein L, Blass J. A double-blind placebo-controlled study of 3,4-diaminopyridine in amytrophic lateral sclerosis patients on a rehabilitation unit. J Neurol Sci 1996;138(1-2):93-6
   PubMed Web of Science ®Google Scholar
• Miller RG, Smith SA, Murphy JR, A clinical trial of verapamil in amyotrophic lateral sclerosis. Muscle Nerve 1996;19(4):511-5
   PubMed Web of Science ®Google Scholar
• Ryberg H, Askmark H, Persson LI. A double-blind randomized clinical trial in amyotrophic lateral sclerosis using lamotrigine: effects on CSF glutamate, aspartate, branched-chain amino acid levels and clinical parameters. Jul Acta Neurol Scand. 2003;108(1):1-8
   PubMed Web of Science ®Google Scholar
• Berto MC, Filha SC, Camelier A, Acute action of aminophylline in patients with amyotrophic lateral sclerosis. Acta Neurol Scand 2007;115(5):301-5 Links 2007;115(5):301-5
   Google Scholar
• Norris FH, Tan Y, Fallat RJ, Elias L. Trial of oral physostigmine in amyotrophic lateral sclerosis. Clin Pharmacol Ther 1993;54(6):680-2
   PubMed Web of Science ®Google Scholar
• Cudkowicz ME, Shefner JM, Schoenfeld DA, Trial of celecoxib in amyotrophic lateral sclerosis. Ann Neurol 2006;60(1):22-31
   PubMed Web of Science ®Google Scholar
• A controlled trial of recombinant methionyl human BDNF in ALS: The BDNF Study Group (Phase III). Neurology 1999;52(7):1427-33
   PubMed Web of Science ®Google Scholar
• Group ACTS. A double-blind placebo-controlled clinical trial of subcutaneous recombinant human ciliary neurotrophic factor (rhCNTF) in amyotrophic lateral sclerosis. Neurol 1996;46:1244-9
   PubMed Web of Science ®Google Scholar
• Lai EC, Felice KJ, Festoff BW, Effect of recombinant human insulin-like growth factor-I on progression of ALS. A placebo controlled study. The North America ALS/IGF-1 Study Group. Neurology 1997;49:1621-30
   PubMed Web of Science ®Google Scholar
• Meininger V, Bensimon G, Bradley WR, Efficacy and safety of xaliproden in amyotrophic lateral sclerosis: results of two Phase III trials. Amyotrop Lateral Scler 2004;5(2):107-17
   Web of Science ®Google Scholar
• Guiloff RJ, Eckland DJ, Demaine C, Controlled acute trial of a thyrotrophin releasing hormone analogue (RX77368) in motor neuron disease. J Neurol Neurosurg Psychiatry 1987;50(10):1359-70
   PubMed Web of Science ®Google Scholar
• Hesselmans LF, Wieneke GH, Oey PL, Evaluation of electrophysiological and clinical tests in an exploratory trial of Org 2766 in motor neuron disease. Neuromuscul Disord 1993;3(4):319-25
   PubMedGoogle Scholar
• Johansson A, Engler H, Blomquist G, Evidence for astrocytosis in ALS demonstrated by [11C](L)-deprenyl-D2 PET. J Neurol Sci 2007;255(1-2):17-22
   PubMed Web of Science ®Google Scholar
• Brody JA, Chen KM, Yase Y, Inosiplex and amyotrophic lateral sclerosis. Therapeutic trial in patients on Guam. Arch Neurol 1974;30(4):322-3
   PubMed Web of Science ®Google Scholar
• Olson WH, Simons JA, Halaas GW. Therapeutic trial of tilorone in ALS: lack of benefit in a double-blind, placebo-controlled study. Neurology 1978;28(12):1293-5
   PubMed Web of Science ®Google Scholar
• Lacomblez L, Bouche P, Bensimon G, Meininger V. A double-blind, placebo-controlled trial of high doses of gangliosides in amyotrophic lateral sclerosis. Neurology 1989;39(12):1635-7
   PubMed Web of Science ®Google Scholar
• Olarte MR, Shafer SQ. Levamisole is ineffective in the treatment of amyotrophic lateral sclerosis. Neurology 1985;35(7):1063-6
   PubMed Web of Science ®Google Scholar
• Appel SH, Stewart SS, Appel V, A double-blind study of the effectiveness of cyclosporine in amyotrophic lateral sclerosis. Arch Neurol 1988;45(4):381-6
   PubMed Web of Science ®Google Scholar
• Poutiainen E, Hokkanen L, Niemi ML, Färkkilä M. Reversible cognitive decline during high-dose alpha-interferon treatment. Pharmacol Biochem Behav 1994 Apr; 47(4):901-5
   PubMed Web of Science ®Google Scholar
• Olarte MR, Gersten JC, Zabriskie J, Rowland LP. Transfer factor is ineffective in amyotrophic lateral sclerosis. Ann Neurol 1979;5(4):385-8
   Google Scholar
• Smith SA, Miller RG, Murphy JR, Ringel SP. Treatment of ALS with high dose pulse cyclophosphamide. J Neurol Sci 1994;124 (Suppl): 84–7
   PubMedGoogle Scholar
• Meyer T, Maier A, Borisow N, Thalidomide causes sinus bradycardia in ALS. Journal of Neurology 2008;255 (4):587-91
   PubMed Web of Science ®Google Scholar
• Beghi E, Chio A, Inghilleri M, A randomized controlled trial of recombinant interferon beta-1a in ALS. Neurology 2000;54(2):469-74
   PubMed Web of Science ®Google Scholar
• Gordon PH, Moore DH, Gelinas DF, Placebo-controlled Phase I/II studies of minocycline in amyotrophic lateral sclerosis. Neurology 2004;62(10):1845-7
   PubMed Web of Science ®Google Scholar
• Louwerse ES, Weverling GJ, Bossuyt PM, Randomized, double-blind, controlled trial of acetylcysteine in amyotrophic lateral sclerosis. Arch Neurol 1995;52:559-64
   PubMed Web of Science ®Google Scholar
• Levy G, Kaufmann P, Buchsbaum R, A two-stage design for a Phase II clinical trial of coenzyme Q10 in ALS. Neurology 2006;66(5):660-3
   PubMed Web of Science ®Google Scholar
• Mazzini L, Testa D, Balzarini C, Mora G. An open-randomized clinical trial of selegiline in amyotrophic lateral sclerosis. J Neurol 1994;241(4):223-7
   PubMed Web of Science ®Google Scholar
• Chiò A, Cucatto A, Terreni AA, Schiffer D. Reduced glutathione in amyotrophic lateral sclerosis: an open, crossover, randomized trial. Ital J Neurol Sci 1998 Dec; 19(6):363-6
   PubMedGoogle Scholar
• Miller R, Bradley W, Cudkowicz ME, Phase II/III randomized trial of TCH346 in patients with ALS. Neurology 2007;69(8):776-84
   PubMed Web of Science ®Google Scholar
• Meininger V, Asselain B, Guillet P, Pentoxifylline in ALS: A double-blind, randomized, multicenter, placebo-controlled trial. Neurology 2006;66(1):88-92
   PubMed Web of Science ®Google Scholar
• Tyler HR. Double-blind study of modified neurotoxin in motor neuron disease. Neurology 1989;39(12):1635-7
   PubMedGoogle Scholar
• Rivera VM, Grabois M, Deaton W, Modified snake venom in amyotrophic lateral sclerosis. Lack of clinical effectiveness. Arch Neurol 1980;37(4):201-3
   PubMed Web of Science ®Google Scholar
• Scelsa SN, MacGowan DJ, Mitsumoto H, A pilot, double-blind, placebo-controlled trial of indinavir in patients with ALS. Neurology 2005;64(7):1298-300
   PubMed Web of Science ®Google Scholar
• Kalmar B, Greensmith L. Heat shock proteins as therapeutic targets in Amyotrophic Lateral Sclerosis. In: Asea AA, Brown IR; eds. Heat Shock Proteins and the Brain: implications for neurodegenerative diseases and neuroprotection. Springer, 2008. P. 69-107
   Google Scholar